Catalyst Pharmaceuticals Announces Enrollment of First Patient in Phase 3 Trial of Firdapse® in MuSK Antibody Positive Myasthenia Gravis
“By conducting this Phase 3 study in patients with MuSK-MG, we hope to provide a potential treatment option for people suffering from this rare condition,” said
“There is a significant unmet medical need to treat the symptoms of MuSK-MG, and these patients are eagerly awaiting a new treatment option,” said
About the MSK-002 Clinical Trial
Catalyst previously announced that it had reached agreement with the
Additional information about MSK-002 can be found on www.clinicaltrials.gov (NCT03304054).
About myasthenia gravis and MuSK-MG
Myasthenia gravis (MG) is a rare, debilitating, autoimmune disease that affects the neuromuscular junction—the place where nerve cells and muscle cells communicate. In MG, the body attacks special receptors on nerve cells that are sensitive to an important chemical called acetylcholine (ACh). ACh is an important chemical because it helps send messages from nerves across the neuromuscular junction to muscles, where these special receptors interpret the message. These special receptors are called acetylcholine receptors (AChR). There is a second chemical that is needed for proper nerve-muscle communication and it is called muscle-specific kinase (MuSK). MuSK also helps nerve-muscle communication across the neuromuscular junction. Unlike AChR-MG, in which the body attacks the receptor, in patients with MuSK-MG, their bodies attack cells that produce the MuSK chemical. It’s estimated that up to 8% of all people with MG (or about 3,000-4,800 total patients in the U.S.) have the MuSK-MG type.
Catalyst is also developing CPP-115 to treat refractory infantile spasms. CPP-115 has been granted U.S. Orphan Drug Designation for the treatment of infantile spasms by the
This press release contains forward-looking statements. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst's actual results in future periods to differ materially from forecasted results. A number of factors, including (i) whether the results of the MSK-002 study will be successful, (ii) the timing of the enrollment of the MSK-002 study, (iii) whether the NDA recently submitted for Firdapse for LEMS will be accepted by the
Chief Executive Officer
Source: Catalyst Pharmaceuticals, Inc.